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WO2023015309A3 - Éditeurs primaires améliorés et leurs procédés d'utilisation - Google Patents

Éditeurs primaires améliorés et leurs procédés d'utilisation Download PDF

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Publication number
WO2023015309A3
WO2023015309A3 PCT/US2022/074628 US2022074628W WO2023015309A3 WO 2023015309 A3 WO2023015309 A3 WO 2023015309A3 US 2022074628 W US2022074628 W US 2022074628W WO 2023015309 A3 WO2023015309 A3 WO 2023015309A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
prime
prime editors
improved
editors
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2022/074628
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English (en)
Other versions
WO2023015309A2 (fr
Inventor
David R. Liu
Peter J. CHEN
Jordan Leigh DOMAN
Smriti PANDEY
Monica NEUGEBAUER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Broad Institute Inc
Harvard University
Original Assignee
Broad Institute Inc
Harvard University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Broad Institute Inc, Harvard University filed Critical Broad Institute Inc
Priority to EP22764605.6A priority Critical patent/EP4381057A2/fr
Priority to JP2024507108A priority patent/JP2024530487A/ja
Priority to CN202280067531.5A priority patent/CN118056010A/zh
Priority to AU2022325166A priority patent/AU2022325166A1/en
Priority to US18/681,490 priority patent/US20250270593A1/en
Priority to CA3227004A priority patent/CA3227004A1/fr
Publication of WO2023015309A2 publication Critical patent/WO2023015309A2/fr
Publication of WO2023015309A3 publication Critical patent/WO2023015309A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)

Abstract

La présente divulgation concerne des compositions et des procédés d'édition primaire présentant une efficacité d'édition améliorée et/ou une formation d'indel réduite à l'aide d'éditeurs primaires modifiés et de protéines de fusion d'éditeur primaire. La divulgation concerne en outre des vecteurs, des cellules et des kits comprenant les compositions et les polynucléotides de la divulgation.
PCT/US2022/074628 2021-08-06 2022-08-05 Éditeurs primaires améliorés et leurs procédés d'utilisation Ceased WO2023015309A2 (fr)

Priority Applications (6)

Application Number Priority Date Filing Date Title
EP22764605.6A EP4381057A2 (fr) 2021-08-06 2022-08-05 Éditeurs primaires améliorés et leurs procédés d'utilisation
JP2024507108A JP2024530487A (ja) 2021-08-06 2022-08-05 改善されたプライムエディターおよび使用方法
CN202280067531.5A CN118056010A (zh) 2021-08-06 2022-08-05 改进的引导编辑器和使用方法
AU2022325166A AU2022325166A1 (en) 2021-08-06 2022-08-05 Improved prime editors and methods of use
US18/681,490 US20250270593A1 (en) 2021-08-06 2022-08-05 Improved prime editors and methods of use
CA3227004A CA3227004A1 (fr) 2021-08-06 2022-08-05 Editeurs primaires ameliores et leurs procedes d'utilisation

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163230688P 2021-08-06 2021-08-06
US63/230,688 2021-08-06
US202263388888P 2022-07-13 2022-07-13
US63/388,888 2022-07-13

Publications (2)

Publication Number Publication Date
WO2023015309A2 WO2023015309A2 (fr) 2023-02-09
WO2023015309A3 true WO2023015309A3 (fr) 2023-03-16

Family

ID=83188605

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2022/074628 Ceased WO2023015309A2 (fr) 2021-08-06 2022-08-05 Éditeurs primaires améliorés et leurs procédés d'utilisation

Country Status (6)

Country Link
US (1) US20250270593A1 (fr)
EP (1) EP4381057A2 (fr)
JP (1) JP2024530487A (fr)
AU (1) AU2022325166A1 (fr)
CA (1) CA3227004A1 (fr)
WO (1) WO2023015309A2 (fr)

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US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
EP3177718B1 (fr) 2014-07-30 2022-03-16 President and Fellows of Harvard College Protéines cas9 comprenant des intéines dépendant de ligands
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
EP3658573A1 (fr) 2017-07-28 2020-06-03 President and Fellows of Harvard College Procédés et compositions pour l'évolution d'éditeurs de bases à l'aide d'une évolution continue assistée par phage (pace)
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2020154500A1 (fr) 2019-01-23 2020-07-30 The Broad Institute, Inc. Protéines chargées supernégativement et utilisations associées
EP3956349A1 (fr) 2019-04-17 2022-02-23 The Broad Institute, Inc. Éditeurs de base d'adénine présentant des effets hors cible réduits
CA3231677A1 (fr) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Procedes et compositions pour moduler un genome
WO2024077267A1 (fr) 2022-10-07 2024-04-11 The Broad Institute, Inc. Méthodes et compositions d'édition d'amorce pour traiter des troubles de répétition de triplet
WO2024240138A1 (fr) * 2023-05-22 2024-11-28 江西农业大学 Système d'édition primaire fondé sur la transcriptase inverse perv
WO2024243415A1 (fr) 2023-05-23 2024-11-28 The Broad Institute, Inc. Éditeurs primaires évolués et ingéniérisés à efficacité d'édition améliorée
WO2025022290A1 (fr) 2023-07-21 2025-01-30 Crispr Therapeutics Ag Modulation de l'expression du gène alas1 (5'-aminolévulinate synthase 1)
WO2025048436A1 (fr) * 2023-08-29 2025-03-06 성균관대학교산학협력단 Éditeur primaire pour corriger le gène eys et utilisation associée
WO2025096936A2 (fr) 2023-11-03 2025-05-08 The Broad Institute, Inc. Utilisation de réécriture par matrice d'arn dans la correction de mutations dans cdkl5
WO2025151814A1 (fr) 2024-01-10 2025-07-17 The Broad Institute, Inc. Utilisation d'édition primaire dans la correction de mutations de cftr
WO2025217616A1 (fr) 2024-04-12 2025-10-16 The Broad Institute, Inc. Édition primaire et édition de base du gène atp1a3 pour le traitement de l'hémiplégie alternante de l'enfant
CN120536504A (zh) * 2025-07-29 2025-08-26 江西农业大学 基于内源性多基因编辑的阿尔茨海默病模型猪的构建方法

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Also Published As

Publication number Publication date
WO2023015309A2 (fr) 2023-02-09
US20250270593A1 (en) 2025-08-28
CA3227004A1 (fr) 2023-02-09
JP2024530487A (ja) 2024-08-21
EP4381057A2 (fr) 2024-06-12
AU2022325166A1 (en) 2024-02-08

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